Treatment of retinitis pigmentosa

Abstract

A polynucleotide comprising a nucleotide sequence encoding the retinitis pigmentosa GTPase regulator ORF15 isoform (RPGR ORF15 ), wherein the RPGR ORF15 -encoding nucleotide sequence has been codon optimised to increase fidelity of replication of the sequence.

1 - 48 . (canceled) 
     
     
         49 . A composition comprising a nucleic acid comprising
 (a) a sequence encoding a rhodopsin kinase promoter, and   (b) a sequence encoding a retinitis pigmentosa GTPase regulator ORF15 isoform (RPGR ORF15 ).   
     
     
         50 . The composition of  claim 49 , wherein the rhodopsin kinase promoter is a GRK1 promoter. 
     
     
         51 . The composition of  claim 49  or  50 , wherein the sequence encoding the promoter has at least 80% identity to the nucleic acid sequence of SEQ ID NO: 7. 
     
     
         52 . The composition of any one of  claims 49  to  51 , wherein the sequence encoding the promoter comprises the nucleic acid sequence of SEQ ID NO: 7. 
     
     
         53 . The composition of any one of  claims 49  to  51 , wherein the sequence encoding the promoter consists of the nucleic acid sequence of SEQ ID NO: 7. 
     
     
         54 . The composition of any one of  claims 49  to  53 , wherein the sequence encoding the RPGR ORF15  is a human RPGR ORF15  sequence. 
     
     
         55 . The composition of any one of  claims 49  to  54 , wherein the sequence encoding the RPGR ORF15  comprises a nucleotide sequence encoding an amino acid sequence that has at least 80% identity to the amino acid sequence of SEQ ID NO: 1. 
     
     
         56 . The composition of any one of  claims 49  to  55 , wherein the sequence encoding the RPGR ORF15  comprises a nucleotide sequence that has at least 80% identity to the nucleic acid sequence of SEQ ID NO: 2. 
     
     
         57 . The composition of  claim 56 , wherein the sequence encoding the RPGR ORF15  comprises a nucleotide sequence that has at least 97% identity to the nucleic acid sequence of SEQ ID NO: 2. 
     
     
         58 . The composition of any one of  claims 49  to  54 , wherein the sequence encoding the RPGR ORF15  comprises a nucleotide sequence that has at least 80% identity to the nucleic acid sequence of SEQ ID NO: 3. 
     
     
         59 . The composition of  claim 58 , wherein the sequence encoding the RPGR ORF15  comprises a nucleotide sequence that has at least 97% identity to the nucleic acid sequence of SEQ ID NO: 3.